The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...

Like the first death reported in March, Sarepta Therapeutics attributed the fatality to a case of acute liver failure following dosing of the Duchenne muscular dystrophy gene therapy, Elevidys ...

Sarepta Therapeutics shares sank to their lowest level in nine years Monday after a second patient taking its Elevidys drug ...

Duchenne muscular dystrophy affects approximately 15,000 people in the United States, primarily boys. The genetic disorder disrupts production of dystrophin, a protein essential for muscle function.

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular dystrophy.

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the ...

Duchenne Muscular Dystrophy Market Size, Epidemiology, In-Market Drugs Sales, Pipeline Therapies, and Regional Outlook ...

Shares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.

TAS-205 showed no significant impact on motor function in patients with Duchenne muscular dystrophy (DMD), highlighting the ongoing search for effective treatments for the rare condition.

Sarepta is facing a shareholder lawsuit over ELEVIDYS-related deaths. Robbins LLP cites safety risks and stock losses.

In its complete response letter, the FDA cited insufficient evidence establish deramiocel's effectiveness for cardiomyopathy associated with Duchenne muscular dystrophy. The decision comes after CBER ...